RVC and UCB collaborate to better understand diseases of excessive bone formation - Veterinary Practice
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RVC and UCB collaborate to better understand diseases of excessive bone formation

Research by the Royal Veterinary College and biotech company UCB will explore the disease causing mechanisms of two severe bone diseases characterised by excessive bone formation, Sclerosteosis and Ankylosing Spondylitis

A new multi-year research project, led by the Royal Veterinary College (RVC), and in partnership with biopharmaceutical company UCB, will investigate potential novel therapeutic options and mechanism of disease progression in the ultra-rare genetic bone disease Sclerosteosis and the inflammatory condition Ankylosing Spondylitis, respectively. Although the cause of each disease is different, parallels can be drawn due to key pathologies being mediated by the excessive bone formation in specific sites. 

Sclerosteosis patients experience widespread bone overgrowth due to the loss of sclerostin expression, the body’s natural brake on bone formation. This can result in severe, and potentially fatal, pressure on the brain due to increases in skull thickness which can only be temporarily resolved through major surgery.

Ankylosing Spondylitis patients experience bone overgrowth, most commonly in the spine, which can lead to pain, bone fusion and deformity. This is a major cause of structural tissue damage which can result in permanent disability.

The team of researchers, led by Senior Lecturer in Translational Skeletal Research at the RVC, Dr Scott Roberts, hope to make major advances in the understanding of these two diseases. Over the next three years the team will test new potential therapeutic options in preclinical models for Sclerosteosis. This is exceptionally important as there is currently no therapeutic approved to control the excess bone formation observed in this disease.

While there are many approved therapeutics for Ankylosing Spondylitis, the control of pathological bone formation is limited. Therefore, the team will also be researching why this bone forms using specialised disease models via human stem cells.

Dr Scott Roberts said: “I am excited to be working closely with UCB and RVC colleagues to investigate why pathological bone is formed in Ankylosing Spondylitis and how excessive bone formation can be controlled in Sclerosteosis.

“This research interaction will allow us to further our understanding of these specific diseases, with our accrued knowledge also likely to be applicable to other conditions of altered bone metabolism.”

Dr Tim Dreyer, postdoctoral researcher and Sclerosteosis patient, said: “As a patient myself, I am thrilled to be part of a collaboration that aims to potentially develop a therapeutic for Sclerosteosis. It is an incredible opportunity to advance our understanding of this ultra-rare bone disease, while providing hope for a small group of patients and their families.”

Dr Sarah Brown, postdoctoral researcher at the RVC, said: “I am excited to have joined the RVC and am looking forward to conducting research to further understand the mechanism of bone pathology in Ankylosing Spondylitis.” 

While bone is a central mediator of disease in Sclerosteosis and Ankylosing Spondylitis, it is worth remembering it also is an incredibly important organ. Indeed, bone tissue in the skeleton provides the body with both support and protection, making it critical to movement and absorbing impact. It is also an important endocrine organ and mineral depot, contributing to organ function via multiple signalling pathways.

Senior Vice President and Head of Discovery Science at UCB, Dr Alistair Henry, said: “We are delighted to partner with the RVC on this important project to better understand disease progression in these two conditions.

“Although very different diseases, both conditions have a painful and progressive impact on those living with them and we hope this project may pave the way for treatment to improve people’s lives.”

Professor Andrew Pitsillides, professor of skeletal dynamics at the RVC, said: “This research gives us hope of making at least two significant impacts; it will allow us to test some of the central principles about how bone formation is controlled and, vitally, to simultaneously seek out a new therapy for a very rare familial disease.”

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